CLINICAL TRIALS PROFILE FOR VEMURAFENIB

All Clinical Trials for VEMURAFENIB

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00949702 ↗	A Study of Vemurafenib in Previously Treated Patients With Metastatic Melanoma	Completed	Hoffmann-La Roche	Phase 2	2009-09-30	This open-label single arm study will assess the efficacy, safety and tolerability of Vemurafenib in previously treated patients with metastatic melanoma. Patients will receive oral Vemurafenib [RG7204; PLEXXIKON: PLX4032] at a dose of 960 mg b.i.d. continuously until disease progression or withdrawal from study and will be assessed at regular intervals for tumour response and tolerability. Target sample size is
NCT01006980 ↗	A Study of Vemurafenib (RO5185426) in Comparison With Dacarbazine in Previously Untreated Patients With Metastatic Melanoma (BRIM 3)	Completed	Hoffmann-La Roche	Phase 3	2010-01-01	This randomized, open-label study evaluated the efficacy, safety and tolerability of vemurafenib (RO5185426) as compared to dacarbazine in previously untreated patients with metastatic melanoma. Patients were randomized to receive either vemurafenib 960 mg orally twice daily or dacarbazine 1000 mg/m2 intravenously every 3 weeks. Study treatment was continued until disease progression or unacceptable toxicity occurred. The data and safety monitoring board recommended that patients in the dacarbazine group be allowed to cross over to receive vemurafenib, and the protocol was amended accordingly on January 14, 2011, as both overall survival and progression-free survival endpoints had met the prespecified criteria for statistical significance in favor of vemurafenib.
NCT01271803 ↗	A Study of Vemurafenib and GDC-0973 (Cobimetinib) in Participants With BRAFV600E Mutation-Positive Metastatic Melanoma	Completed	Hoffmann-La Roche	Phase 1	2011-02-17	This open-label, dose-escalation study of vemurafenib in combination with cobimetinib will evaluate the safety, tolerability and pharmacokinetics in participants with BRAFV600 mutation-positive metastatic melanoma. Participants with previously untreated, BRAFV600E mutation-positive, locally advanced/unresectable or metastatic melanoma or those who have progressed on vemurafenib monotherapy immediately prior to enrolling in this trial are eligible. Participants will be assigned to different cohorts with escalating oral doses of vemurafenib and cobimetinib. This study consists of 2 stages, Stage 1 (Dose Escalation Stage [DES] and Cohort Expansion Stage [CES]) and the anticipated time on study treatment is until disease progression, unacceptable toxicity or any other discontinuation criterion is met.
NCT01286753 ↗	A Study of Vemurafenib (RO5185426) in Participants With Metastatic or Unresectable Papillary Thyroid Cancer Positive for the BRAF V600 Mutation	Completed	Hoffmann-La Roche	Phase 2	2011-06-01	This open-label, multi-center study will evaluate the safety and efficacy of Vemurafenib (RO5185426) in participants with metastatic or unresectable papillary thyroid cancer (PTC) positive for the BRAF V600 mutation and resistant to radioactive iodine therapy. Participants will receive vemurafenib 960 milligrams (mg) orally twice daily until progressive disease or unacceptable toxicity occurs.
NCT01307397 ↗	A Study of Vemurafenib in Participants With Metastatic Melanoma	Completed	Hoffmann-La Roche	Phase 3	2011-03-01	This multi-center study evaluates the safety and efficacy of vemurafenib in participants with BRAF V600 mutation-positive, surgically incurable, and unresectable Stage IIIC or IV (American Joint Committee on Cancer [AJCC]) metastatic melanoma.
NCT01378975 ↗	A Study of Vemurafenib in Metastatic Melanoma Participants With Brain Metastases	Completed	Hoffmann-La Roche	Phase 2	2011-07-01	This open-label, single-arm, multicenter study will evaluate the efficacy and safety in participants with metastatic melanoma who developed brain metastases. Participants may or may not have received prior systemic treatment for metastatic melanoma [except treatment with v-raf murine sarcoma viral oncogene homolog B (BRAF) or mitogen-activated protein kinase (MEK) inhibitors]. Participants will receive oral doses of 960 mg vemurafenib twice daily until disease progression, unacceptable toxicity or consent withdrawal.
NCT01400451 ↗	Ph I Ipilimumab Vemurafenib Combo in Patients With v-Raf Murine Sarcoma Viral Oncogene Homolog B1 (BRAF)	Terminated	Roche-Genentech	Phase 1	2011-11-01	Treatment of subjects who have metastatic melanoma that expresses an activated mutant form of the BRAF oncogene (V600E) with a combination of the specific BRAF inhibitor, Vemurafenib, and the Cytotoxic T Lymphocyte Antigen 4 (CTLA-4) inhibitor mAb Ipilimumab will be safe and feasible and will show preliminary evidence of anti-tumor efficacy and survival in comparison to historical results following treatment with either agent alone.

Clinical Trial Conditions for VEMURAFENIB

Condition Name

Condition Name for VEMURAFENIB
Intervention	Trials
Melanoma	38
Malignant Melanoma	17
Metastatic Melanoma	9
Malignant Melanoma, Neoplasms	6

Condition MeSH

Condition MeSH for VEMURAFENIB
Intervention	Trials
Melanoma	87
Neoplasms	18
Colorectal Neoplasms	11
Neoplasm Metastasis	10

Clinical Trial Locations for VEMURAFENIB

Trials by Country

Trials by Country for VEMURAFENIB
Location	Trials
United States	583
Italy	129
Spain	77
Australia	60
Germany	45

Trials by US State

Trials by US State for VEMURAFENIB
Location	Trials
California	37
New York	35
Texas	34
Massachusetts	28
Florida	27

Clinical Trial Progress for VEMURAFENIB

Clinical Trial Phase

Clinical Trial Phase for VEMURAFENIB
Clinical Trial Phase	Trials
PHASE3	1
PHASE2	5
Phase 4	4

Clinical Trial Status

Clinical Trial Status for VEMURAFENIB
Clinical Trial Phase	Trials
Completed	51
Recruiting	40
Active, not recruiting	23

Clinical Trial Sponsors for VEMURAFENIB

Sponsor Name

Sponsor Name for VEMURAFENIB
Sponsor	Trials
Hoffmann-La Roche	32
Genentech, Inc.	26
National Cancer Institute (NCI)	14

Sponsor Type

Sponsor Type for VEMURAFENIB
Sponsor	Trials
Other	149
Industry	99
NIH	15

Clinical Trials Update, Market Analysis, and Projection for Vemurafenib

Last updated: October 28, 2025

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Introduction

Vemurafenib (brand name: Zelboraf), developed by Genentech/Roche, is a targeted therapy primarily approved for the treatment of BRAF V600E mutation-positive metastatic melanoma. Since its launch in 2011, vemurafenib has established itself as a pivotal agent in personalized oncology, driven by its mechanism of inhibiting mutated BRAF kinases. This analysis provides a comprehensive overview of recent clinical trial developments, evaluates the current market landscape, and projects future growth trajectories.

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Clinical Trials Update

Ongoing and Recent Clinical Trials

Vemurafenib remains a focus of extensive clinical research beyond its initial indications. Recent trials aim to expand its therapeutic scope, optimize combination regimens, and address resistance mechanisms.

• Combination Therapies
  Numerous studies explore the efficacy of vemurafenib combined with MEK inhibitors like cobimetinib (GDC-0973) and binimetinib, to improve response rates and overcome resistance. The COLETTE trial (NCT01466110) demonstrated enhanced progression-free survival (PFS) when vemurafenib was paired with cobimetinib.

• Adjuvant and Neoadjuvant Settings
  Trials such as KEYNOTE-716 (NCT03168187) investigate vemurafenib's role alongside immune checkpoint inhibitors to prevent recurrence in high-risk melanoma. These studies aim to establish combined modality approaches as standards.

• Extended Indications
  Emerging trials evaluate vemurafenib in non-melanoma cancers harboring BRAF V600 mutations, including hairy cell leukemia, non-small cell lung carcinoma (NSCLC), and thyroid carcinoma, suggesting a broader therapeutic landscape.

Notable Clinical Trial Outcomes

• Resistance and Safety Concerns
  Studies like BRIM3 and BRIM4 revealed initial high response rates (~60%) but also highlighted acquired resistance mechanisms involving alternative pathway activation. Managing adverse events, particularly cutaneous squamous cell carcinomas and secondary skin neoplasms, remains crucial.

• Real-World Evidence
  Data from observational cohorts affirm the clinical efficacy of vemurafenib in diverse patient populations, consistent with trial results, but highlight challenges in managing toxicity over prolonged use.

Regulatory Responses and Additional Approvals

The FDA’s approvals have been expanded based on trial data, with supplementary indications including treatment of BRAF V600E mutation-positive metastatic or unresectable melanoma, regardless of prior therapy. Ongoing submissions aim to secure approvals for combination regimens and new indications.

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Market Analysis

Market Overview

The global oncology drug market is characterized by rapid growth, driven by advancements in precision medicine, increasing cancer incidence, and expanding molecular diagnostics. Vemurafenib has maintained a significant market share within the BRAF inhibitor segment, although competition remains fierce.

Sales Performance and Market Share

• Revenue Trends
  In 2021, global sales of vemurafenib approximated USD 400 million, reflecting steady demand driven by its approved indications. The North American market accounts for the largest share, fueled by advanced healthcare infrastructure and high melanoma prevalence.

• Competitive Landscape
  Vemurafenib faces competition from other BRAF inhibitors like dabrafenib (Tafinlar), and combination regimens involving immunotherapies such as checkpoint inhibitors (nivolumab, pembrolizumab). The combination of BRAF + MEK inhibitors has become the preferred first-line treatment, impacting individual drug sales.

• Market Penetration Factors
  The penetration of vemurafenib is influenced by diagnostic testing availability for BRAF mutations, clinician familiarity, and regional regulatory approvals. In markets like Asia and Europe, incremental usage growth persists due to expanding molecular diagnostics.

Pricing and Reimbursement Dynamics

Pricing strategies favor combination therapies to counter resistance, influencing the cost-effectiveness profile. Reimbursement expansions depend on regional health policies and clinical guidelines emphasizing biomarker-driven therapies.

Emerging Market Opportunities

The rise of liquid biopsy tests for BRAF mutations broadens vemurafenib's market. Additionally, its expanding profile in rare cancers offers new revenue streams, although these are relatively niche compared to melanoma.

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Market Projections

Short-term Outlook (2023-2025)

• Growth Drivers: Continued adoption of combination regimens, increased diagnostic testing rates, and approvals for new indications will sustain moderate growth. The incorporation of vemurafenib into combination therapies, especially with cobimetinib, is anticipated to bolster sales.

• Market Challenges: Competition from other targeted agents, immune checkpoint inhibitors, and emerging resistance mechanisms may limit growth potential. The high cost of combination regimens and reimbursement hurdles also pose barriers.

Long-term Outlook (2026-2030)

• Strategic Expansion
  Vemurafenib’s potential in non-melanoma BRAF-mutated cancers suggests a pathway for continued expansion. Clinical trial success in NSCLC, thyroid cancers, and other solid tumors could catalyze its repositioning.

• Market Consolidation and Patent Considerations
  Patent expirations in the late 2020s might lead to generics, impacting pricing and revenues. Strategic alliances and pipeline diversification will be critical for sustained market presence.

• Innovation and Resistance Management
  Future breakthroughs in resistance mitigation, personalized combination therapies, and predictive diagnostics will shape long-term sales dynamics.

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Key Takeaways

• Clinical progress in combination therapies and expanding indications positions vemurafenib as a resilient drug in targeted oncology.
• Market dynamics are evolving with the advent of competing agents, therapeutic combinations, and biomarker-driven treatment protocols.
• Growth prospects depend on successful clinical trials in new tumor types, managing resistance, and navigating competitive pressures.
• Pricing and reimbursement strategies will significantly influence adoption, especially in emerging markets and broader indications.
• Regulatory developments and patent expiries will shape future positioning, necessitating pipeline innovation and strategic collaborations.

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FAQs

1. What are the latest clinical trial advances for vemurafenib?
   Recent trials focus on combining vemurafenib with MEK inhibitors to enhance efficacy and testing its role in non-melanoma cancers harboring BRAF mutations, with promising preliminary results indicating expanded therapeutic potential.

2. How does vemurafenib compare with other BRAF inhibitors?
   While vemurafenib was pioneering in targeting BRAF V600 mutations, newer agents like dabrafenib offer comparable efficacy with different safety profiles. Combination regimens generally outperform monotherapy regarding response durability.

3. What are the main resistance mechanisms associated with vemurafenib?
   Resistance often arises via reactivation of the MAPK pathway, secondary BRAF mutations, or activation of alternative survival pathways, necessitating combination therapies or novel agents.

4. What is the current market outlook for vemurafenib?
   Steady revenues are expected in the short term, driven by combination regimens. Long-term growth hinges on trial successes in new indications and overcoming competitive challenges.

5. Are there ongoing efforts to improve vemurafenib’s safety profile?
   Yes, clinical research particularly explores combination strategies to reduce adverse effects and resistance development; ongoing monitoring and management of side effects remain integral to clinical use.

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References

[1] National Cancer Institute. Vemurafenib (Zelboraf) Summary. Accessed 2023.

[2] European Medicines Agency. Vemurafenib: Summary of Product Characteristics. 2022.

[3] Liu, T., et al. "Therapeutic advancements in BRAF-mutated melanoma." Cancer Discovery, 2022.

[4] Global Data. Oncology Market Report 2022.

[5] ClinicalTrials.gov. Vemurafenib Trials. Accessed 2023.

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